Targeting a specific gene to treat a type of leukemia
Official title: Therapeutic targeting of an oncogenic translational program in AML
Funded at Versiti Wisconsin, INC. · NIH-11132428 · Data updated 2026-11-12
This study is testing a new treatment called AI-10-49 for people with acute myeloid leukemia who have a specific genetic change that makes their condition harder to treat, and it aims to help these patients live longer by killing leukemia cells and working better with other medications.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Versiti Wisconsin, INC. NIH-funded |
| Lab location | 1 site (Milwaukee, United States) |
| Project start | April 1, 2025 |
| Project end | March 31, 2030 |
| Funding year | 2025 |
| Award (this year) | $545,170 |
| NIH project number | 1R01CA293588-01A1 |
| Project ID | NIH-11132428 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults diagnosed with acute myeloid leukemia characterized by the inv(16) genetic alteration.
✕Less likely to help
Patients with acute myeloid leukemia who do not have the inv(16) mutation may not benefit from this specific treatment approach.
What this research studies
This research focuses on a specific genetic alteration in acute myeloid leukemia (AML) known as inv(16), which leads to poor patient outcomes. The team has developed a small molecule inhibitor called AI-10-49 that disrupts the interaction between the oncogene CBFβ-SMMHC and a critical transcription factor, RUNX1. By inducing cell death in leukemia cells, this treatment aims to improve survival rates for patients with this specific genetic mutation. The research also explores combining AI-10-49 with another inhibitor to overcome drug resistance, enhancing the effectiveness of the therapy.
Why it matters
Potential benefit: If successful, this research could provide a targeted therapy that significantly improves survival rates for patients with inv(16) acute myeloid leukemia.
How similar studies have gone: Previous research has shown promising results with targeted therapies in leukemia, indicating potential for success with this novel approach.
Where this research is happening
-
Versiti Wisconsin, INC.
Milwaukee, United States Active
Researchers
- Principal investigator: Pulikkan, John — Versiti Wisconsin, INC.
- Study coordinator: Pulikkan, John
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.